AAV1-hOTOF gene therapy for autosomal recessive deafness 9: a single-arm trial-Reference-Cited by-同舟云学术

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AAV1-hOTOF gene therapy for autosomal recessive deafness 9: a single-arm trial

Published:2024-05 Issue:10441 Volume:403 Page:2317-2325
ISSN:0140-6736
Container-title:The Lancet
language:en
Short-container-title:The Lancet

Author:

Lv Jun,Wang Hui,Cheng Xiaoting,Chen Yuxin,Wang Daqi,Zhang Longlong,Cao Qi,Tang Honghai,Hu Shaowei,Gao Kaiyu,Xun Mengzhao,Wang Jinghan,Wang Zijing,Zhu Biyun,Cui Chong,Gao Ziwen,Guo Luo,Yu Sha,Jiang Luoying,Yin Yanbo,Zhang Jiajia,Chen Bing,Wang Wuqing,Chai Renjie,Chen Zheng-Yi,Li Huawei,Shu Yilai

Funder

MEEI

Shanghai Municipal Health Commission

National Natural Science Foundation of China

Ministry of Science and Technology of the People's Republic of China

Fudan University

Shanghai Municipal Education Commission

Science and Technology Commission of Shanghai Municipality

Publisher

Elsevier BV

Reference39 articles.

1. Newborn hearing screening—a silent revolution;Morton;N Engl J Med,2006

2. Global, regional, and national incidence, prevalence, and years lived with disability for 354 diseases and injuries for 195 countries and territories, 1990-2017: a systematic analysis for the Global Burden of Disease Study 2017;Spencer;Lancet,2018

3. Otoferlin, defective in a human deafness form, is essential for exocytosis at the auditory ribbon synapse;Roux;Cell,2006

4. Comprehensive genetic testing in the clinical evaluation of 1119 patients with hearing loss;Sloan-Heggen;Hum Genet,2016

5. A multicenter study on the prevalence and spectrum of mutations in the otoferlin gene (OTOF) in subjects with nonsyndromic hearing impairment and auditory neuropathy;Rodríguez-Ballesteros;Hum Mutat,2008

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1. A free intravesicular C-terminal of otoferlin is essential for synaptic vesicle docking and fusion at auditory inner hair cell ribbon synapses;Progress in Neurobiology;2024-09

2. Novel PTPRQ variants associated with hearing loss in a Chinese family PTPRQ variants in Chinese hearing loss;Frontiers in Genetics;2024-08-14

3. A base editor for correcting deafness;Nature Biomedical Engineering;2024-08-12

4. A base editor for the long-term restoration of auditory function in mice with recessive profound deafness;Nature Biomedical Engineering;2024-08-12

5. Toward Optogenetic Hearing Restoration;Annual Review of Neuroscience;2024-08-08

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